Fibrosis Project Q&A

We recently announced £82,405 funding to investigate the development of a potential new test system for drugs that impact fibrosis in DMD. We have put together a short Q&A to explain why this project is so important and what it could bring to the Duchenne community.

Q: Why is this project so important?

A: There are currently no anti-fibrotic therapies approved specifically for the treatment of DMD. We need to understand better the fibrosis that occurs in the heart and skeletal muscle in people with DMD to allow us to discover therapeutic targets and develop therapies.

Drug development is a long and expensive process. Many potential treatments fail at the clinical phase because they do not work in the same way in early clinical trials as predicted by pre-clinical research. Part of the reason for this is that most pre-clinical work is currently carried out using animal models of human disease.By investing in,and improving the relevance of, pre-clinical research we hope toincrease the chance of accurately predicting how treatments will work in patients. By using test systems morerepresentative of the human disease, for example using human tissue, we mayreduce the likelihood of failure at the clinical stages.

This project aims to investigate the possibility of creating slices of human cardiac tissue and maintaining their viability in vitro. Systems such as this have been developed for some tissues, such as the liver and lung, but not in heart tissue.

Q: What will the outcomes of this project be?

A: This pilot study aims to see if it is possible to develop a human cardiac precision cut slice (PCS)test system for target identification and validation in DMD. Put simply, we aim to see if it is possible to create thin slices of human cardiac tissue to use as a model system to test anti-fibrotic and anti-inflammatory drugs for DMD. The project has 3main aims:

Determine the optimalconditions to maintain the viability of the slices.

Investigate how we can use the cardiac slices to model the inflammation and fibrosis seen in DMD.

Finally, test a range of known anti-inflammatory and anti-fibrotic drugs to determine the potential value of the model as a test system.

Q: How will this project benefit the Duchenne community?

A: The overall aim of this project is to create a model which mimics the cardiac tissues in people with DMD with the specific aim of testing anti-fibrotics in future. Using this system to test potential new treatments in pre-clinical research should provide us with more relevant results than using animal models of the disease. Compounds tested in functional disease-mimicking tissues is more likely to be effective in early clinical trials. This should improve the landscape of anti-fibrotic drug discovery and help improve the lives of boys affect by Duchenne muscular dystrophy

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